Autoimmune Hyperthyroidism in Prepubertal Children With Onset Before 6 Years of Age: Characteristics at Diagnosis and Follow-Up Trends.
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Number of clinical symptoms at diagnosis
研究概览
简要总结
The investigators propose a multicenter retrospective study to assess clinical, biochemical, and auxological characteristics at diagnosis and during follow-up in a cohort of Caucasian pediatric patients diagnosed with autoimmune hyperthyroidism before puberty. These prepubertal patients will be compared with a control group of post-pubertal patients with Graves' disease.
This study aims to enhance the understanding of autoimmune hyperthyroidism in prepubertal patients by providing a detailed evaluation of disease onset, therapeutic response, and growth-related outcomes. The inclusion of a carefully matched post-pubertal control group will allow for robust comparative analysis and identification of age-dependent clinical patterns and prognostic indicators, ultimately supporting more tailored and effective management strategies in pediatric populations at this particular age.
详细描述
There are only few studies in literature focusing on the analysis of cohorts of patients with autoimmune hyperthyroidism with onset exclusively in early childhood. The investigators therefore propose this study with the aim of retrospectively assessing characteristics at onset and during follow-up in a cohort of Caucasian hyperthyroid subjects diagnosed with the disease before puberty.
OBJECTIVES AND ENDPOINTS
- Primary Objective To evaluate the clinical and biochemical features at the onset of autoimmune hyperthyroidism in patients diagnosed in prepubertal age Secondary Objectives
- To evaluate of biochemical parameters during medical therapy
- To evaluate of auxological parameters at diagnosis and during follow-up
- To compare the previous data with the clinical and biochemical features at diagnosis and during the follow-up in patients diagnosed with Graves during the pubertal age (> Tanner 2)
Inclusion Criteria 1. Subjects with autoimmune hyperthyroidism 2. Prepubertal stage (Tanner 1) at diagnosis Exclusion Criteria 3. Subjects with hyperthyroidism not of autoimmune aetiology 4. Pubertal stage (>Tanner 2) at diagnosis
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects with autoimmune hyperthyroidism
排除标准
- •Subjects with hyperthyroidism not of autoimmune aetiology
研究组 & 干预措施
50 patients affected by autoimmune hyperthyroidism in prepubertal age
干预措施: restrospective observational study (Other)
50 patients affected by autoimmune hyperthyroidism in pubertal age
干预措施: restrospective observational study (Other)
结局指标
主要结局
Number of clinical symptoms at diagnosis
时间窗: Day 0
Total number of symptoms per patient, categorized as none (0), mild (1-2), moderate (3-5), or severe (\>5).
Number of clinical signs of hyperthyroidism at diagnosis
时间窗: Day 0 (retrospective assessment at diagnosis)
Total number of clinical signs (including goiter, exophthalmos, and tachycardia) present at diagnosis, recorded as a count per patient.
Serum free triiodothyronine (FT3) levels at diagnosis
时间窗: Day 0
Measurement of serum FT3 (pmol/L) at diagnosis.
Serum free thyroxine (FT4) levels at diagnosis
时间窗: Day 0
Measurement of serum FT4 (pmol/L) at diagnosis.
Serum thyroid-stimulating hormone (TSH) levels at diagnosis
时间窗: Day 0
Measurement of serum TSH (mIU/L) at diagnosis.
Serum TSH receptor antibody (TRAb) levels at diagnosis
时间窗: Day 0
Measurement of TRAb levels (IU/L) at diagnosis.
Thyroid autoantibody positivity at diagnosis (TPOAb and TgAb)
时间窗: Day 0
Presence or absence of thyroid autoantibodies (TPOAb and TgAb), reported as positive/negative.
次要结局
- Aauxological parameters at diagnosis and during follow-up(day 0, diagnosis; at 6 months; at 12 months)
- Duration of antithyroid drug therapy(Baseline (treatment initiation) to treatment discontinuation (up to 24 months))
- Remission rate after antithyroid drug therapy(At 24 months after treatment initiation)
- Rate of definitive treatment (thyroidectomy or radioactive iodine therapy)(Baseline to 24 months)
- Serum TSH receptor antibody (TRAb) levels at time of the definitive treatment(At time of definitive treatment (up to 24 months))
- Time to normalization of thyroid function(Baseline to normalization (up to 12 months))
- Number of antithyroid drug discontinuation attempts(Baseline to 24 months)
- Annual growth velocity during follow-up(Baseline to 24 months)
- Bone age during follow-up(At baseline, 12 months, and 24 months)
研究者
Vigone Maria Cristina
medical doctor - Specialist in Pediatrics Expert in Pediatric Endocrinology Head of the Thyroid Pathology Outpatient Clinic
IRCCS Ospedale San Raffaele